House lawmakers, facing pressure from more than 150 patient and rare disease advocacy organizations, put back into their latest Cures draft a measure to grant an additional six months of exclusivity for approved drugs that receive a new indication for a rare disease or condition, after having dropped the language from the second draft of the bill unveiled in late-April. The Orphan Product Extensions Now (OPEN) Act was the only exclusivity provision that made its way back into the updated draft released Wednesday (May 13), after all the exclusivity language was stripped in the second version. Some patient groups thought a provision offering 15-years exclusivity for “dormant therapies” would also reappear and were disappointed it didn't.
The latest Cures draft released a day ahead of a scheduled House Energy and Commerce health subcommittee markup offers some tweaks and provisions to the second draft of the bill put out by E&C chair Fred Upton (R-MI) and Rep. Diana DeGette (D-CO), who have led the Cures initiative.
The first draft of Cures legislation released in January included a number of provisions increasing exclusivity for a wide variety of drugs. But all of the exclusivity provisions were dropped in the second draft released April 29, following push back from the generic drug lobby, consumer groups and other stakeholders who were worried increased exclusivity would slow access to cheaper generics.
The second draft, however, included a place holder section titled "Repurposing Drugs for Serious and Life-Threatening Diseases and Conditions" that many stakeholders felt would contain compromise measures on extending exclusivity to spur drug development, including some version of the dormant therapies language from the January draft of the Cures bill.
As lawmakers and staff negotiated the latest draft ahead of Thursday's markup, patient and rare disease advocates made a major lobbying effort through a “day of action” Tuesday (May 12) to make sure the OPEN Act was included, Julia Jenkins, executive director of EveryLife Foundation for Rare Diseases, told Inside Health Policy Wednesday.
“We all united together in a day of action yesterday to have advocates call their member of Congress and ask them to make sure OPEN was included in the legislation that was going to be introduced,” Jenkins said.
Jenkins said the OPEN Act was modeled on the Best Pharmaceuticals for Children Act, which provides six months exclusivity for doing a study on pediatric diseases. She said that act was extremely successful, resulting in 400 labeling changes for new indications for pediatric diseases on existing drugs.
“For orphan drugs there are less than 500 approved treatments for rare diseases, so if we could have a success of 400 new drugs on label for rare diseases we would essentially double the available treatments.”
Under the OPEN Act provisions a drug could get up to a year of additional exclusivity for a new indication if that indication also treats pediatric cancer, because new pediatric cancer drugs already get an additional six-months exclusivity under the Biologics Price Competitions and Innovation Act.
Other stakeholders like the National Health Council (NHC) were disappointed that exclusivity provisions for dormant therapies weren't included in the latest cures draft, after expecting to see some form of compromise language in the measure.
“The patient advocacy community thanks Committee Chairman Fred Upton (R-MI) and Committee Member Diana DeGette (D-CO) for their tireless work to present a bipartisan bill. However, we believe the Committee has missed a huge opportunity to include a game-changing dormant therapies provision that could have revolutionized the development of treatments desperately needed by people with long-term, life-threatening conditions.,” said Mark Boutin, CEO of NHC.
“Though we are extremely disappointed that the Committee did not include dormant therapies in the draft legislation, the patient advocacy community will continue to call for an open, honest, public debate on incentivizing the creation of new treatments. Too many men, women, and children have died waiting for cures for us to give up now,” Boutin added.
Following release of the January draft of Cures legislation, the Generic Pharmaceutical Association (GPhA) lashed out hard against exclusivity provisions, particularly the dormant therapies provision.
"[T]he Dormant Therapies provision of the bill would potentially grant brand drug companies an unprecedented increase in exclusivity for a curiously broad category of new drugs, delaying the competition from generic drugs and biosimilars that promotes beneficial innovations in treatments," Ralph Neas, GPhA president and CEO, said in the Jan. 27 press statement.
GPhA did not issue a statement following Wednesday's version of Cures legislation, but said it may have one soon after the group reviews the latest iteration.
The E&C health subcommittee will consider the newest version Thursday, with the goal of getting legislation to the full committee in the near future.
“Every step in this process has been hard fought, not for our own interests, but for those who suffer. Every policy has been scrutinized, every voice considered. We’ve pushed harder and faster than anyone thought possible, because we know patients cannot wait. They need 21st Century Cures now,” Upton said in a press release Wednesday. -- Todd Allen Wilson (twilson@iwpnews.com)